On July 26, 2019, the Platelet Disorder Support Association (PDSA) hosted the first Externally-led Patient-Focused Drug Development (EL-PFDD) meeting on Immune Thrombocytopenia (ITP) to share with officials at the U.S. Food and Drug Administration (FDA), and other key stakeholders, including industry and research institutions, the perspectives of people living with immune thrombocytopenia (ITP). The panel of disease experts included patients and caregivers who shared the impact of ITP on their daily lives, mental health and overall quality of life. They expressed their expectations and priorities for current and future treatments for ITP.
PDSA hosted the meeting as part of ITP Conference 2019, which convened more than 300 attendees in our Nation’s Capital. The subsequent "Voice of the Patient: Immune Thrombocytopenia" report is a compilation of the testimonies and survey responses of individuals affected by ITP from all around the world and is the first-ever report of its kind for this rare autoimmune bleeding disorder.
PDSA would like to gratefully acknowledge the many individuals who contributed to the outstanding success of this meeting, especially the caregivers, patients and family members who courageously shared their journeys, clinicians and researchers who shared scientific insight, and industry representatives, and leaders from the FDA who listened with thoughtfulness and intent. The 2019 Immune Thrombocytopenia EL-PFDD and the Voice of the Patient report represent important milestones for the ITP community; it is our hope that this effort and Voice of the Patient report will inform and guide both drug development and regulatory efforts such that the significant unmet therapeutic needs of people living with immune thrombocytopenia is better understood and satisfied.
The Voice of the Patient report is a critical resource for researchers in development and submission of new therapies to the FDA in the regulatory approval process. It will also advise the clinical trial endpoints that are crucial to the well-being of our ITP community.
To learn more about PDSA’s EL-PFDD Meeting, visit the 2019 ITP Conference page.
Learn more about the Patient-Focused Drug Development (PFDD) initiative, visit the FDA Patient-Focused Drug Development meetings page.
2019 PDSA EL-PFDD Meeting - Part 1
2019 PDSA EL-PFDD Meeting - Part 2
“It was a great meeting. Your group was extremely well organized and I think very helpful for those with the disease. Thank you for inviting me.”
Ann T. Farrell, MD
Division of Nonmalignant Hematology
Office of Cardiology, Hematology, Endocrinology, and Nephrology
Office of New Drugs
Center for Drug Evaluation and Research